FDA Approves BridgeBio's Attruby (acoramidis) for ATTR-CM

Key Insights

• The FDA has approved Attruby (acoramidis) from BridgeBio, marking the first new treatment in over five years for transthyretin amyloid cardiomyopathy (ATTR-CM).
• ATTR-CM, affecting over 400,000 globally, results from misfolded protein deposits in the heart, leading to impaired function and, without treatment, death within five years of diagnosis.
• Attruby aims to stabilize transthyretin, preventing misfolding and subsequent amyloid formation, offering a new therapeutic option for both genetic and age-related ATTR-CM.

The Food and Drug Administration (FDA) has approved BridgeBio's Attruby (acoramidis) for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). This approval marks a significant milestone as the first new treatment option for this progressive heart disease in over five years.

ATTR-CM is a condition characterized by the misfolding and aggregation of transthyretin protein, leading to amyloid deposits in the heart. These deposits impair heart function, and without treatment, most patients succumb to the disease within five years of diagnosis. The disease affects an estimated 400,000 people worldwide and can arise from genetic mutations or aging.

Acoramidis, the active ingredient in Attruby, is designed to stabilize the transthyretin protein, preventing its misfolding and subsequent amyloid formation. By addressing the underlying cause of ATTR-CM, Attruby offers a potential disease-modifying approach to treatment.

This approval represents BridgeBio's first major commercial product, addressing a critical unmet need in the treatment of ATTR-CM. The availability of Attruby provides a new therapeutic option for patients suffering from this debilitating and life-threatening condition.